Enrico Costa, Vittorio Del Grosso, Bernadette Cappello, Armando A Genazzani, Benedikt Huttner, Hubert G M Leufkens, Nicola Magrini, Francesco Nonino, Veronika J Wirtz, Hendrika A van den Ham, Lorenzo Moja
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引用次数: 0
Abstract
Background: The WHO Model List of Essential Medicines (EML) includes those medicines that offer the best health payback for individuals and health systems. It serves as a guide for countries to develop and update national EMLs. The implementation of essential medicines policies is therefore mostly oriented to medicines on the EML. However, medicines evaluated and not recommended for inclusion in the EML also have relevant implications for development of efficient medicine policies. This study analyzed the characteristics, frequencies, and reasons for applications for medicines proposed for inclusion in the WHO EML not being recommended.
Methods: Assessment of the recommendations for all medicines proposed for inclusion in the WHO EML in reports of the Expert Committee on Selection and Use of Essential Medicines in the WHO Technical Reports Series from 2002 to 2023. We collected key information from EML applications including active substance, therapeutic indication, orphan status, applicant, and reasons for negative recommendations. Logistic univariate and multivariate regression analyses assessed predictive characteristics for applications with negative recommendations.
Results: A total of 359 applications for addition of new medicines to the EML were submitted: 211 (58.8%) received a positive recommendation. Among the 148 (41.2%) applications with a negative recommendation, the most prevalent reasons for not recommending were quality of clinical evidence (62.1%) and economic criteria (33.1%). Concerns about capacity to implement the new medicines in health care systems or requiring specialized expertise increased over time. Applications submitted by pharmaceutical companies, individuals not affiliated with scientific societies or non-governmental organizations, and academia were more prone to receiving a negative recommendation.
Discussion: An appreciable proportion of applications for addition of new medicines to the EML are not recommended. Over time, low or limited quality of clinical evidence was a consistent explanatory reason leading to non-recommending. Economic considerations and feasibility are emerging justifications for non-recommending.
期刊介绍：
Frontiers in Medicine publishes rigorously peer-reviewed research linking basic research to clinical practice and patient care, as well as translating scientific advances into new therapies and diagnostic tools. Led by an outstanding Editorial Board of international experts, this multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to researchers, academics, clinicians and the public worldwide.
In addition to papers that provide a link between basic research and clinical practice, a particular emphasis is given to studies that are directly relevant to patient care. In this spirit, the journal publishes the latest research results and medical knowledge that facilitate the translation of scientific advances into new therapies or diagnostic tools. The full listing of the Specialty Sections represented by Frontiers in Medicine is as listed below. As well as the established medical disciplines, Frontiers in Medicine is launching new sections that together will facilitate
- the use of patient-reported outcomes under real world conditions
- the exploitation of big data and the use of novel information and communication tools in the assessment of new medicines
- the scientific bases for guidelines and decisions from regulatory authorities
- access to medicinal products and medical devices worldwide
- addressing the grand health challenges around the world
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